title: Regression of subependymal giant cell astrocytoma with rapamycin in tuberous sclerosis complex.
abstract: The authors present a 21-year-old woman who has been receiving rapamycin for 5 months for bilateral subependymal giant cell astrocytomas. The patient was started at a dose of 0.2 mg/kg/day. Levels were maintained between 11 and 13 ng/mL. Magnetic resonance imaging of the brain 2(1/2) months after initiating rapamycin demonstrated a decrease in size of both astrocytomas (11 to 7.5 mm on the right and 8 to 5 mm on the left). Further studies are needed with prolonged observation to confirm these findings, determine the length of necessary treatment, and evaluate recurrence risk after discontinuation of rapamycin.
authors.affiliation: Department of Pediatrics, Division of Child and Adolescent Neurology, The University of Texas Health Science Center, Houston, Texas, USA. mary.k.koenig@uth.tmc.edu
authors.forename: Mary Kay
authors.initials: MK
authors.lastname: Koenig
authors.forename: Ian J
authors.initials: IJ
authors.lastname: Butler
authors.forename: Hope
authors.initials: H
authors.lastname: Northrup
mesh_terms: D000903:Antibiotics, Antineoplastic / Q000008:administration & dosage*; D001921:Brain / Q000187:drug effects / Q000473:pathology; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology; D004305:Dose-Response Relationship, Drug; D005260:Female; D018315:Glioma, Subependymal / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology; D006801:Humans; D008279:Magnetic Resonance Imaging; D020123:Sirolimus / Q000008:administration & dosage*; D016896:Treatment Outcome; D014402:Tuberous Sclerosis / Q000188:drug therapy* / Q000473:pathology / Q000503:physiopathology; D055815:Young Adult
chemical_list: D000903:Antibiotics, Antineoplastic; D020123:Sirolimus